ADPD 2025: Unlocking GLP-1's potential in neurodegeneration
At the recently launched Alzheimer’s & Parkinson’s Diseases Conference held in Vienna, Lotte Bjerre Knudsen from Novo Nordisk A/S, who has extensive experience in glucagon-like peptide-1 (GLP-1) research, delivered a plenary lecture focused on the role of GLP-1 receptor agonists, such as semaglutide, in attenuating neuroinflammation and neurodegeneration.

ADPD 2025: Unlocking GLP-1's potential in neurodegeneration
At the recently launched Alzheimer’s & Parkinson’s Diseases Conference held in Vienna, Lotte Bjerre Knudsen from Novo Nordisk A/S, who has extensive experience in glucagon-like peptide-1 (GLP-1) research, delivered a plenary lecture focused on the role of GLP-1 receptor agonists, such as semaglutide, in attenuating neuroinflammation and neurodegeneration.
Airna’s $155M series B advances RNA editing drug AIR-001 for AATD
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
A nanoparticle-based 'cocktail' vaccine against atherosclerosis
In a study published in Nature Communications, researchers from Nanjing University present a novel nanovaccine cocktail formulated by individual conjugation of antigen p210 and adjuvant CpG ODNs onto superparamagnetic iron oxide nanoparticles (SPIONs).
Multiomic Health and Alloy Therapeutics to partner on renal tissue-targeting drugs
Multiomic Health Ltd. and Alloy Therapeutics Inc. have signed a memorandum of understanding to jointly discover and develop first-in-class renal tissue-targeting drugs. Precision medicines for chronic renal conditions targeted to the kidney tissue of specific patient subpopulations are expected to improve efficacy and safety compared to conventional systemic drugs.
Other news to note for April 2, 2025
Additional early-stage research and drug discovery news in brief.
Cryptic pocket in CB1 receptor is better target for analgesia
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced.
FORCE platform boosts muscle function in dystrophic mice
Dyne Therapeutics Inc. presented their most recent work to advance novel therapeutics for facioscapulohumeral muscular dystrophy (FHSD). FSHD is an autosomal dominant genetic disorder characterized by muscle weakness and atrophy. Dyne Therapeutics previously developed the FORCE platform, which uses an antigen-binding fragment (Fab) specifically targeting telomeric repeat binding factor-1 (TfR1) for targeted therapeutic delivery.
Syndax Pharmaceuticals patents new prodrugs for viral infections
Syndax Pharmaceuticals Inc. has disclosed extended purine tricyclic and bicyclic nucleosides as prodrugs reported to be useful for the treatment of viral infections.

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Conferences

FORCE platform leads to Duchenne muscular dystrophy mouse model
Neurology/psychiatric
Dyne Therapeutics Inc. is advancing novel therapeutics for people living with genetically driven neuromuscular diseases. Representatives from the company recently presented a poster showcasing preclinical and early clinical results from the development of their proprietary FORCE platform applied to...
Repare Therapeutics details discovery of PLK4 inhibitor RP-1664
Cancer
The synthesis of RP-1664, a highly potent, selective and bioavailable PLK4 inhibitor for the potential treatment of cancer, was recently reported by Repare Therapeutics Inc.
Prelude Therapeutics reports preclinical profile of first-in-human SMARCA2 degrader
Cancer
Prelude Therapeutics Inc. described the discovery of PRT-3789, a first-in-human, highly potent and selective SMARCA2-targeted protein degrader, for the potential treatment of cancer.
FAP inhibitor AZD-2389 is a strong candidate for MASH treatment
Endocrine/metabolic
Fibroblast activation protein (FAP) is a serine protease, the expression of which increases with pathogenic fibroblasts in the fibrotic liver during metabolic dysfunction-associated steatohepatitis (MASH) and might induce fibrosis by cleaving several proteins that regulate extracellular matrix...

Cancer

Navigo Proteins and SCK CEN collaborate to advance next-generation radiotheranostics
Collaboration
Navigo Proteins GmbH has established a strategic research collaboration with SCK CEN, the Belgian Nuclear Research Centre, to advance the preclinical development of next-generation radiotheranostics using the Auger-electron emitting radioisotope Terbium (161Tb).

Infection

Cell-penetrating bispecific HBcAg/pre-S1 antibody suppresses HBV replication and secretion
Bispecific antibody
Hepatitis B virus (HBV) infection is associated with liver diseases, including chronic hepatitis, which notably increases the risk of cirrhosis and hepatocellular carcinoma (HCC) development. Although some of the current treatment strategies promote virological suppression, they are insufficient to...

Institut Pasteur advances Lassa fever vaccine based on new MOPEVAC platform
Immune
An Institut Pasteur team has developed an original vaccine platform known as MOPEVAC, that will strengthen the organization’s pandemic preparedness initiatives, with the platform’s first vaccine candidate, which targets Lassa fever, set to enter the clinic.
Combining antibody domain, NA-targeting drug makes for broad-spectrum antibody
Immune
The ability of influenza virus to rapidly undergo antigenic shift to evade immunity raises the need for effective influenza antivirals with a broad spectrum. In a recent Nature Microbiology article, Cidara Therapeutics Inc. provided preclinical data for their drug-Fc conjugate compound CD-388,...

Neurology/psychiatric

Ono and Reborna partner to discover RNA-targeting small molecules for neurological disorders
Drug design, drug delivery and technologies
Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with Reborna Biosciences Inc. to generate RNA-targeting novel small molecules in the field of the central nervous system.
Dissecting the role of neural circuits in mental health
Conferences
In the brain, molecular information is transmitted between cells through neural circuits. Synapses establish connections between the pathways that run from one area to another, allowing the most complex organ in the body to fulfill different functions. Cells and neural circuits are the basic...
Roche describes new SARM1 inhibitors for neurodegenerative disorders
Patents
Researchers from F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have presented NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Dock7-mutant zebrafish show disrupted skeletal muscle myofiber structure and reduced locomotion
Conferences
Previous studies have shown that protein expression of DOCK7 is increased in skeletal muscle biopsies from patients with Duchenne muscular dystrophy (DMD), leading researchers from the University of Alabama at Birmingham and affiliated organizations to assess the functional impact of DOCK7 on...
AL-001 enhances brain-specific lithium delivery, minimizes systemic exposure in preclinical Alzheimer’s disease
Conferences
Alzamend Neuro Inc. recently presented pharmacokinetic data for the lithium-based sparing therapy AL-001, a lithium salicylate/L-proline co-crystal, in 5XFAD mice, a murine model of Alzheimer’s disease (AD).

Endocrine/metabolic

Novo Nordisk licenses Lexicon’s preclinical obesity drug in $1B deal
Deals and M&A
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals...

Biomarkers

Rare genetic variants in HECTD2 tied to bipolar disorder
Neurology/psychiatric
A variant burden analysis for bipolar disorder was performed using gene-based aggregation of loss-of-function variants in whole-genome sequencing data from Iceland and the UK Biobank; the association between bipolar disorder and the burden of loss-of-function variants was tested in 13,786 genes.

PRTFDC1 expression predicts prognosis, drug response of testicular germ cell tumors
Cancer
Researchers from Guangdong Medical University published data from a study that aimed to identify novel biomarkers that would allow for comprehensive assessment of prognosis and immunotherapy response in patients with testicular germ cell tumors (TGCT).
Increased HKDC1 and phosphorylated RBBP5 emerge as potential targets and prognostic markers in HCC
Cancer
Metabolic reprogramming in cancer involves glycolytic enzymes acquiring noncanonical functions and acting as protein kinases, which contribute to tumor progression and present new therapeutic opportunities. While hexokinase domain-containing protein 1 (HKDC1), a hexokinase family member, has been...

Gastrointestinal

ABHD17B identified as a target in liver fibrosis
RNAi
Scientists at Massachusetts General Hospital have revealed how chronic liver injury alters hepatic stellate cells (HSCs), triggering the formation of fibrotic scarring in the liver. The researchers have shown that this cell type...
Unlocking early genetic drivers of stomach cancer
Cancer
Research published online in Nature on March 19, 2025, closely examines the changes occurring in the gastric epithelium during the progression toward cancer development. Certain mutations that occur in normal, nonreproductive cells over time can...
ICB treatment induces colitis-related indicators in DSS drinking mouse model
Conferences
Researchers from Chinese Academy of Sciences detailed the creation of a new dextran sulfate sodium (DSS)-based mouse model of immune checkpoint blockade (ICB) therapy-associated colitis.
Domain Therapeutics nominates biased antagonist of PAR2 as drug candidate for inflammatory diseases
Dermatologic
Domain Therapeutics SA has nominated PAR2 antagonist DT-9046 as a drug candidate with potential to treat various inflammatory diseases, including atopic dermatitis, inflammatory bowel disease and arthritis, as well as neuroinflammatory conditions...
Hepatic stellate cells shape and control liver function
Researchers at the German Cancer Research Center Deutsches Krebsforschungszentrum (DKFZ) and their collaborators have cast new light on the mechanisms by which hepatic stellate cells control liver metabolism and regeneration. The work builds on...
Targeting NAT10 as an approach for IBD management
Immune
Inflammatory bowel disease (IBD) is impacted by genetic, environmental and immunological factors, where the imbalance in T-cell immune responses significantly promotes its progression. In recent years, the role of RNA modifications in epigenetic...

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